FDA Updates for the Week of Aug. 21, 2023

FDA Approves First Biosimilar of MS Drug Tysabri

The FDA has approved Sandoz’s Tyruko (natalizumab-sztn), the first biosimilar to Biogen’s Tysabri (natalizumab) injection to treat adults with relapsing forms of multiple sclerosis (MS). Developed by Polpharma Biologics, Tyruko, like Tysabri, is also indicated for inducing and maintaining clinical response and remission in adult patients with moderately to severely active Crohn’s disease.

Tyruko is expected to be available as soon as possible. A Sandoz spokesperson said launch timing will be dependent on other factors, including FDA approval of its JCV assay (a test to detect the level of antibodies for a virus that can be carried to the brain), as well as having HCPCS code in place. The spokesperson would not comment on the price at this point.

The prescribing information for both Tyruko and Tysabri contains a boxed warning about the increased risk of progressive multifocal leukoencephalopathy, a viral infection of the brain that usually leads to death or severe disability. Because of this risk, both products are available under a risk evaluation and mitigation strategy.

The approval of Tyruko was based on data from the phase 3 Antelope study conducted by Polpharma Biologics. The Antelope study met its primary endpoint, showing comparable efficacy for cumulative combined unique active (CUA) lesions. Together with the phase I (PK/PD) study, which also met its primary endpoint, the studies demonstrated that the Tyruko biosimilar matched the efficacy and safety of the reference medicine.

FDA Approves Abrysvo to Prevent RSV in Infants

The FDA has approved Pfizer’s Abrysvo vaccine to prevent respiratory syncytial virus infections in infants through the age of six months. It is the first vaccine approved to be given to pregnant women at 32 to 36 weeks to prevent infection in infants. Abrysvo is administered as a single dose injection into the muscle.

Pfizer’s RSVpreF is a bivalent vaccine candidate that is composed of equal amounts of recombinant RSV prefusion F from subgroups A and B. The FDA approved Abrysvo in May for the prevention of RSV in people 60 years of age and older.

The FDA’s decision is based on the data from the pivotal phase 3 clinical trial MATISSE, a placebo-controlled study designed to evaluate the efficacy, safety, and immunogenicity of the vaccine against lower respiratory tract disease due to RSV in infants born to healthy individuals vaccinated during pregnancy.

FDA Approves Ingrezza for Disorder Associated with Huntington’s Disease

The FDA has approved Ingrezza (valbenazine) capsules to treat adults with chorea associated with Huntington’s disease, a progressive neurodegenerative disorder. Most people with Huntington’s disease experience chorea, an involuntary movement disorder that is characterized by irregular and unpredictable movements.

Developed by Neurocrine Biosciences, Ingrezza is a once-daily selective vesicular monoamine transporter 2 (VMAT2) inhibitor. It is believed to reduce extra dopamine signaling, which may lead to fewer uncontrollable movements. Ingrezza is also approved to treat tardive dyskinesia. It has a list price of $8,457 for a supply of 28 capsules, according to Drugs.com. A spokesperson said the new approval will not effect its price.

Ingrezza is covered for more than eight out of 10 patients nationwide, according to a Neurocrine spokesperson. The company’s Inbrace support program helps patients with access. Patients with commercial insurance may be eligible for copay assistance and patients with no prescription coverage may be eligible for participation in the patient assistance program.

FDA Approves Higher Dose Eylea

The FDA has approved Eylea HD (aflibercept) injection 8 mg to treat patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR). Eylea HD is administered every four weeks for the first three months across all indications, followed by every eight to 16 weeks in wAMD and DME and every eight to 12 weeks for diabetic retinopathy.

Developed by Regeneron and Bayer, Eylea HD has a list price of $2,625 per single-use vial, according to a Regeneron statement. The company is launching Eylea HD early this week. A spokesperson said patients who have Medicare Part B will have immediate coverage for approved indications. Patients with commercial insurance are expected to have coverage in the next six months.

Eylea 2 mg is already available with a wholesale acquisition cost of $1,957.55, according to Drugs.com. In addition, Eylea is available as a 0.4 mg dose to treat retinopathy of prematurity.

FDA Sets Review Date for First-in-Class Therapy for MDS

The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of June 16, 2024, for Geron’s new drug application (NDA) for imetelstat, a first-in-class treatment to treat transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS). In addition, the FDA informed the company that it is currently planning to hold an advisory committee meeting as part of the NDA review.

Myelodysplastic syndromes are a group of cancers in which blood stem cells do not mature. This can lead to anemia, infection or bleeding. Transfusions help manage systems and can slow progression to leukemia.

Imetelstat targets telomerase to inhibit uncontrolled proliferation of malignant stem and progenitor cells in myeloid hematologic malignancies. Telomerase is a naturally occurring enzyme that maintains telomeres, a protein structure at both ends of each chromosome that protects the chromosome from becoming frayed. Each time a cell divides, the telomeres become shorter; telomerase stops this from happening.

Geron submitted an NDA in June 2023 based on data from the phase 3 study IMerge to evaluate imetelstat versus placebo. The primary endpoint of transfusion independence at eight weeks was met for imetelstat-treated patients (39.8%) vs. placebo (15.0%). Key secondary endpoint of 24-week transfusion independence. Imetelstat-treated patients also experienced a clinically meaningful mean reduction in red blood cell transfusions compared with placebo.

FDA Grants Priority Review to Xtandi for Earlier Treatment in Prostate Cancer

The FDA has accepted and granted priority review for the supplemental new drug application (sNDA) for Xtandi (enzalutamide) to treat patients with non-metastatic castration-sensitive prostate cancer with an increasing prostate-specific antigen (PSA) level, which is referred to biochemical recurrence (BCR). The Prescription Drug User Fee Act (PDUFA) date for an anticipated FDA decision is in fourth quarter of 2023.

Developed by Astella and Pfizer, Xtandi is an androgen receptor signaling inhibitor. It is already available to treat patients with castration-resistant prostate cancer and metastatic castration-sensitive prostate cancer. The list price of Xtandi 40 mg is $14,383 for a supply of 120 capsules, according to Drugs.com. The companies offer copay support, as well as a patient assistance program for those who are uninsured.

The submission is based on results from the phase 3 EMBARK trial. The study met its primary endpoint of metastasis-free survival for the Xtandi plus leuprolide arm, demonstrating a 58% reduction in the risk of metastasis or death over placebo plus leuprolide. The most common adverse events with the combination therapy were fatigue, hot flush, and arthralgia and in those treated with Xtandi monotherapy were fatigue, gynecomastia, and arthralgia.